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Orphan Drug Markets in Europe

NEW YORK, Dec. 20, 2011 /PRNewswire/ -- announces that a new market research report is available in its catalogue:

Orphan Drug Markets in Europe

Key developments and the strategies of major pharmaceutical companies

The successful introduction of orphan products in Europe remains problematic for the developers, although they can seek to alleviate some of the issues by carefully developing a launch strategy.

The first marketing authorisations for orphan drugs in Europe took place a decade ago. Since then there have been over 60 designated orphan products. These products now account for annual sales of more than $6 billion.

Traditionally the development and marketing of orphan drugs has not been an area of interest for major pharmaceutical companies, reflecting the view that orphan drugs were specialty products with limited market potential thus offering a poor fit for companies based upon the blockbuster model. However, a number of major companies have reassessed their strategy with respect to orphan drugs and five of the top ten have made explicit commitments to the development of such drugs. This has seen a number of changes in the orphan drug landscape and further developments are likely, especially from some of those not yet active in the field.

The 302 drugs that are approved for the treatment of 373 orphan indications only over a fraction of the approximately 7,000 rare diseases categorised as rare by the National Institute for Health (US).

The products Glivec and Tracleer show, it is possible for orphan drugs to generate substantial returns in Europe clearly demonstrating the commercial viability of Europe as a market for orphan drugs.

Orphan Drug Markets in Europe - Key developments and the strategies of major pharmaceutical companies

 is a unique report from URCH Publishing that offers the reader a comprehensive overview of the state of the orphan medicines market in Europe. The carefully authored study provides expert insight into the market to date, regulatory challenges and future opportunities concerning orphan drugs. Particular attention is paid to the differences in regulation and market between Europe Japan and the US.

Some key findings from the report include:

-Within Europe sales of approved orphan products account for approximately $6bilion.-Novartis' Glivec (imatinib mesylate) sales are over $1.5 billion.-Since January 2000 there has been a steady increase in applications for orphan designation with the Committee for Orphan Medicinal Products (COMP) averaging ten positive recommendations per month.-Five companies generated major revenues from the marketing of orphan drug products in 2010, four primarily by supplying biological products while just Actelion supplied small molecule therapeutics.-Big pharma has started to take orphan drugs seriously and a number have formed dedicated business units to explore opportunities.-Alexion's Soliris shows that small patient populations are not incompatible with commercial success.-Although central approval covers 30 European countries, it does not necessarily provide for national availability as each national authority has to agree labelling and reimbursement.-The increasing collaboration between the FDA and EMA has led to the adoption of a common application form should facilitate developers' efforts to exploit orphan drugs on a more international basis.

Reasons to Buy

-Obtain a complete understanding of the orphan drug regulations in Europe.-Gain insight into the key companies operating in this sector.-Understand the differences between the Europe and US markets.-Evaluate which rare diseases offer the greatest commercial opportunity.-Assess the performance of specialist orphan drug companies.-Review how big pharma is starting to enter this market segment.-Find the new areas of pharmaceutical market growth and key opportunities for delivering successful sales growth over the next five years.-Support internal planning and decision-making with an external perspective founded on detailed analysis and transparent market forecasts.

Who should read this unique report

This report is designed for anyone who needs a comprehensive overview of orphan medicines in Europe, including:-Strategy managers and directors in large pharmaceutical companies-Biotechnology companies with niche product pipelines-Investors looking for opportunities-US-based companies with an interest in expanding to Europe.

Companies & Organisations Mentioned in the Report Include

Actelion, Alexion, Alnara Pharmaceuticals, Amgen, Amicus Therapeutics, AmpliPhi Biosciences, Aventis, Bayer, BiogenIdec, Biovitrum, bluebird bio, Bristol-Myers Squibb, Celgene, Cephalon (Teva), COMP, Del Mar Pharmaceuticals, Edimer Pharmaceuticals, Eli Lilly, EURODIS, EUSA Pharma, FDA, Généthon, GlaxoSmithKline, Hyperion Therapeutics, Intermune, Isis Pharmaceuticals, JCR Pharmaceutical, Lotus Tissue Repair, Merck, National Institute for Health, NORD, Novartis, Novo Nordisk, Orfagen, Orphan Europe (Recordati), Orphan Therapeutics, Pfizer, Pharmamar, Prosena, Protalix Biotherapeutics, Roche, Sanofi, Santhera Pharmaceuticals,   Shire, Sigma-Tau, SpePharm, Swedish Orphan Biovitrum, Sygnis Bioscience, Synageva BioPharma, Takeda, Teva, Trophos, United Therapeutics, Vivendy Therapeutics, Voisin Consulting, Zacharon Pharmaceuticals, Zymenex

Orphan medicines mentioned in this report include

Adcirca – Aldurazyme – Avonex – Campath -  Cerezyme - ClolarCopaxone - Elaprase - Epogen - Esbriet – Evoltra - ExJade – Fabrazyme - Gleevec / Glivec – Kogenate - Mozobil - Myozyme (and Lumizyme) – Neupogen – Nexavar – Novoseven - Provigil – Pulmozyme - Rebif – Revatio – Revlimid – Rituxan – Sensipar – Soliris - Somavert  - Sprycel – Temodar – Thyrogen – Tracleer – Velcade – Veletri – Ventavis – Xagrid – Yondelis - Zavesca

Executive Summary Chapter 1: An introduction to orphan drugs in Europe


Historical Overview

Chapter 2: Orphan Drug LegislationGlobal progressThe United StatesEuropeCOMP


Other markets





South Korea


A comparison

Chapter 3: Rare Diseases and Orphan DrugsOverviewRare DiseasesGenetic disordersCystic fibrosisMuscular dystrophyThalassaemiaLysosomal storage disordersCancerVery Rare DiseasesRepurposingPatient registries

Chapter 4: Progress of orphan drugs in Europe


Revenues from orphan drugs in Europe

What is similar?

Designated orphan drugs

Leading applicants

Success stories

Soliris (Alexion)

Enzyme replacement therapies


Glivec (Novartis)

Fragmented market issues

Chapter 5: Orphan drugs in the USOverview25 years of approvalsMajor successes

Chapter 6: Specialist Companies


Major players






Established specialist players

Orphan Europe (Recordati)

Sigma-Tau SpA

Swedish Orphan Biovitrum


EUSA Pharma


Emerging speciality companies

Amicus Therapeutics

AmpliPhi Biosciences

bluebird bio

Del Mar Pharmaceuticals

Edimer Pharmaceuticals


Hyperion Therapeutics

Lotus Tissue Repair

Orphan Therapeutics


Santhera Pharmaceuticals


Sygnis Bioscience

Synageva BioPharma


Vivendy Therapeutics



Chapter 7: Major pharmaceutical companies' involvement in orphan drugsIntroductionEli LillyGlaxoSmithKlineNovartisPfizerSanofiOther companiesOutlook

Chapter 8: Outlook for orphan drugs

Targeting rare diseases more popular

High profile

Commercial opportunities

Potential pitfalls

Competition issues

European specific issues




List of Figures Figure 2.1 Global Time Line of Orphan Drug Legislation

Figure 2.2 FDA definitions of similarity

Figure 2.1 EMA key relationships relating to the designation and approval of orphan drugs

Figure 2.2 EU regulations pertaining to the orphan drug regulation (Regulation 141/2000)

Figure 2.3 The process of orphan drug designations by the COMP

Figure 2.4 The number of applications to the COMP for orphan drug designation, and their outcomes, to July 2011

Figure 3.1 Relative prevalence of 27 lysosomal storage disorders in Australia

Figure 4.1 Orphan drug authorizations in Europe by year (to July 2011)

Figure 4.2 Orphan drug approvals by marketing company

Figure 4.5 Approved orphan drugs by therapeutic area

Figure 6.4 Potential return from an orphan therapeutic in Europe for a prevalent indication

Figure 4.7 Applications for Orphan Drug Designation in Europe

Figure 4.8 Unsuccessful Applications for Orphan Drug Designation in Europe

Figure 4.9 COMP opinions by patient population

Figure 4.10 Geographic availability of 21 orphan drugs in Europe in 2007

Figure 4.11 Confirmed availability, and unavailability, of 60 orphan drugs in selected EU countries in 2007

Figure 4.12 Average price and reimbursement delays for generic products by country

Figure 4.13 Intermune's proposed EU launch strategy for Esbriet

Figure 5.14 European orphan designations by disease prevalence to 2010

Figure 5.15 New Drug Approvals by the FDA 2004-2010

Figure 7.1 The changing orphan drug landscape and major pharma

Figure 8.1 Competitive developments for treating Gaucher disease

Figure 8.2 Availability of 21 approved orphan drugs in Europe in 2007

Figure 8.3 Intermune's European launch strategy for Esbriet

List of Tables Table 2.1 Comparison of Orphan Drug Criteria in the US, the EU and Japan

Table 4.1 European status of therapies for pulmonary hypertension

Table 5.2 Selected commercially successful biological orphan products in the US

Table 5.3 Selected commercially successful small molecule orphan products in the US

Table 6.4 Genzyme's orphan drug portfolio and 2010 sales

Table 6.5 Genzyme's orphan drug pipeline

Table 6.6 Actelion's orphan drug portfolio and 2010 sales

Table 6.7 Actelion's orphan drug pipeline

Table 6.8 Shire's orphan drug portfolio and 2010 sales

Table 9.6 Shire's orphan drug pipeline

Table 6.10 BioMarin's orphan drug portfolio and 2010 sales

Table 6.11 BioMarin's orphan drug pipeline

Table 6.12 Recordati's orphan drug portfolio

Table 6.13 Recordati's orphan drug pipeline

Table 6.11 Sigma-Tau's orphan drug pipeline

Table 6.12 Swedish Orphan Biovitrum's orphan drug portfolio and 2010 revenues

Table 6.13 PharmaMar's orphan drug pipeline

Table 6.14 Orfagen's orphan drug pipeline

Table 6.15 Amicus' orphan drug pipeline

Table 6.16 Prosensa's orphan drug pipeline

Table 6.17 Santhera's orphan drug pipeline

Table 6.18 Synagis' orphan drug pipeline

Table 6.14 Synageva's orphan drug pipeline

To order this report:Drug and Medication Industry: Orphan Drug Markets in Europe


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Nicolas Bombourg
US: (805)652-2626
Intl: +1 805-652-2626





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